WASHINGTON, Aug. 30 (Xinhua) -- The U.S. Food and Drug Administration (FDA) on Wednesday approved the country's first gene therapy that reengineers people's own immune cells to treat childhood leukemia.
In a statement, the FDA called the approval "a historic action," saying it will usher in "a new approach to the treatment of cancer and other serious and life-threatening diseases."
"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," said FDA Commissioner Scott Gottlieb.
"New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses."
The U.S. regulator approved Novartis's Kymriah (tisagenlecleucel), formerly known as CTL019, for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
Kymriah is a novel immunotherapy using an individual patient's own T-cells, a type of white blood cell known as a lymphocyte.
The patient's T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein, known as a chimeric antigen receptor or CAR, that directs the T-cells to target and kill leukemia cells that have a specific antigen, CD19, on the surface.
Once the cells are modified, they are infused back into the patient to kill the cancer cells.
"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," said Peter Marks, director of the FDA's Center for Biologics Evaluation and Research.
"Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials."
The safety and efficacy of Kymriah were demonstrated in one multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The overall remission rate within three months of treatment was 83 percent.
Acute lymphoblastic leukemia is a cancer of the bone marrow and blood, in which the body makes abnormal lymphocytes.
The disease progresses quickly and is the most common childhood cancer in the U.S., with about 3,100 patients aged 20 and younger being diagnosed each year.
Patients often undergo multiple treatments including chemotherapy, radiation, targeted therapy or stem cell transplant, yet less than 10 percent of patients survive five years.
Acute lymphoblastic leukemia can be of either T- or B-cell origin, with B-cell the most common.
Kymriah is approved for use in pediatric and young adult patients with B-cell acute lymphoblastic leukemia and is intended for patients whose cancer has not responded to or has returned after initial treatment, which occurs in an estimated 15-20 percent of patients.
"Five years ago, we began collaborating with the University of Pennsylvania (Penn) and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need," Joseph Jimenez, CEO of Novartis, said in a statement.
"With the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care," said Jimenez.
Carl June, director of the Center for Cellular Immunotherapies in Penn's Perelman School of Medicine, who is a pioneer of this new treatment, called the approval "a significant step forward in individualized cancer treatment that may have a tremendous impact on patients' lives.
"Through our collaboration with Novartis, we are creating the next wave of immunocellular cancer treatments, and are eager to progress CAR-T therapy in a host of hematologic and other cancer types," June said.
